Imetelstat, a telomerase inhibitor, is capable of depleting myelofibrosis stem and progenitor cells.

ABSTRACT: Clinical trials of imetelstat therapy have indicated that this telomerase inhibitor might have disease-modifying effects in a subset of patients with myelofibrosis (MF). The mechanism by which imetelstat induces such clinical responses has not been clearly elucidated. Using in vitro hematopoietic progenitor cell (HPC) assays and in vivo hematopoietic stem cell (HSC) assays, we examined the effects of imetelstat on primary normal and MF HSCs/HPCs. Treatment of CD34⁺ cells with imetelstat reduced the numbers of MF but not cord blood HPCs (colony-forming unit-granulocyte/macrophage, burst-forming unit-erythroid, and colony-forming unit-granulocyte/erythroid/macrophage/megakaryocyte) as well as MF but not normal CD34⁺ALDH⁺ cells irrespective of the patient's mu

SUBMITTER: Wang X 

PROVIDER: S-EPMC6156882 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature